The medical equipment sector is characterized by a large share of overall health budgets spent for the provision of capital investment goods such as medical scanners and radiotherapy units. A high variability in provision and utilization rates of medical equipment can be observed too. The objective for this study was to contribute to effective cross-border cooperation between European Union (EU)-Member States by pooling resources for high-cost medical equipment investments (1).

Potential cost-intensive and highly specialised medical equipment, where cross-border investment resource pooling may be recommended, were identified by a combined evidence search and expert consultation. An efficiency assessment of medical equipment potential savings for EU-countries was done by a benchmark-approach and a best-practice-approach. Furthermore six examples for cross-border cooperation were investigated and two surveys have been conducted.

The following medical equipment can be considered as cost-intensive and highly specialized across EU-Member States: Magnetic Resonance Imaging (MRI) scanners, Computed Tomography (CT) scanners, Stereotactic systems and Surgical robots.

The efficiency assessment using the benchmark approach was performed for MRI, CT scanners, Positron Emission Tomography (PET) scanners, Angiography units, Gamma cameras and Lithotriptors. The results of the best-practice approach showed potential cost savings due to under-or overutilization per device group and EU-Member State. However, as this analysis offers a view on health systems on a very macro level it was not possible to give detailed insights at the country-level.

The six selected cross-border examples demonstrated a wide variety of options regarding the structure, extent and organization of cross-border cooperation: Five of six cross-border examples were cooperation close to the border, in four of six examples EU funds played an important role.

The study highlighted that cross-border cooperation in the field of cost-intensive/highly specialized medical equipment could bring economic advantages for many EU-Member States. Despite this, still only little is done by EU-Member States in terms of cooperation. Reasons are diverse and can be ascribed to lacking information, differences of national health systems, organizational and administrative hurdles, and lacking political support.

The changing regulatory landscape brings new challenges to Health Technology Assessment (HTA). Marketing authorizations are being granted as the evidence base evolves to facilitate timely patient access to promising health technologies. Consequently, some products come to HTA bodies sooner in their development cycles with less evidence, which ultimately leads to greater uncertainty in decision making. A key challenge for payer and HTA bodies is providing access to promising medicines while the evidence is still emerging, in a financially sustainable way.

Changes to the Cancer Drugs Fund (CDF) have resulted in a managed access fund for cancer medicines in England. The National Institute for Health and Care Excellence (NICE) can now recommend a treatment for use within the CDF if there is plausible potential to satisfy the criteria for routine use in the National Health Service (NHS) at its current price, but the evidence is not robust enough and associated with significant uncertainty. Further evidence is then generated in clinical trials, through observational data collection, or a combination of the two, while the drug's price reflects the decision uncertainty. At the end of the managed access period, NICE reviews the guidance to determine if the treatment can be recommended for routine commissioning.

The first treatment recommended for use within the new CDF was osimertinib for non-small cell lung cancer (1). At the time of NICE appraisal, there was considerable uncertainty in osimertinib's clinical and cost effectiveness because only short-term phase II trial results were available. NICE's independent appraisal committee considered there was plausible potential for osimertinib to be cost effective and identified that an ongoing phase III trial would provide longer-term data addressing the key uncertainties.

An integrated approach between payer and HTA decision-maker has significantly changed how cancer treatments in England are appraised. This collaborative way of working heralds a more sustainable approach to introducing promising cancer treatments.

As of July 2016, funding from England's Cancer Drugs Fund (CDF) is dispensed based on the results of National Institute for Health and Care Excellence (NICE) technology appraisal guidances instead of independent CDF appraisals (1). As part of this transition, NICE is reconsidering drugs previously funded through the CDF (2). This analysis examines CDF reconsiderations conducted between the inception of the new process in July and the end of 2016 to identify any possible trends.

We collected all NICE final technology appraisal guidances (3) completed before the end of 2016 and noted whether each drug was a CDF reconsideration, what the final decision was, and which factors impacted the decisions.

We identified twenty-one NICE oncology reviews competed between July 2016 and the end of 2016. Of these reviews, eight were reconsiderations of drugs previously funded through the old CDF; the rest were new reviews. Only one drug evaluated in the reconsiderations received a negative decision. All the reconsiderations included confidential manufacturer discounts and all noted updated clinical data. End of life (EOL) criteria expanded the acceptable incremental cost-effectiveness ratio (ICER) range for some of the CDF reconsiderations.

All the reconsiderations included updated clinical data and analyses, though it does not appear that updated clinical data were sufficient to bring ICERs to acceptable levels. This is to be expected as the old CDF process served as an alternate funding source for many drugs that did not or were unlikely to fare well under NICE's evaluations. The updated clinical data may have at least increased NICE's confidence in the accuracy of the ICERs. All of the reconsiderations included confidential manufacturer discounts to reach acceptable ICER ranges. The results of this first round of reconsiderations suggest that manufacturers prefer offering their drugs at lower prices to potentially losing National Health Service (NHS) reimbursement entirely.

Faced with increasing financial challenges to the single-payer social security system and constitutional challenges supporting all citizen's right to health, Costa Rica has endeavored to introduce Health Technology Assessment (HTA) to ensure sustainability and promote the timely introduction of technology innovations in the health system. The Ministry of Health initiated a process to establish an independent, external institution providing leadership in the process of HTA.

Based on a survey developed by REDETSA/PAHO (HTA Network of the Americas/Pan American Health Organization), an inclusive method of stakeholders participation was used to analyze the strengths, weaknesses, opportunities and threats regarding the implementation of an HTA entity. This was combined with qualitative research methods, market access situation analysis and the review of coverage and provision processes to define the elements for the new HTA institution. The “in-depth” interviews extended to manufacturers, ministry representatives, services providers, purchasers, patients and citizens representatives, judiciary court, professional colleges, academia and non-governmental organizations (NGOs). Analysis of the professional competencies required for the HTA institution was carried out based on best practice analysis of international HTA institutions.

The implementation of an HTA unit in Costa Rica was identified by all the actors as crucial to ensuring the health system's sustainability. Costa Rica's health system is based on all citizens right to health and all inputs required delivering health services, judicialization and access to health care have become a big issue. Two main issues were identified as essential to implement an HTA institution: the establishment of a clear framework to provide legal and financial support and the need to have sufficient independence from the Ministry and the Social Security, including maximum transparency and methodological robustness.

The business model for the new HTA institution should consider the participation of all the interested actors. The HTA institution should bridge the gap between technology regulation and health technology management and aim to improve both processes. It should also provide third party independent evidence to inform the constitutional court around health care claims.

Rapid technological innovation is leading to new health technologies and interventions becoming available to healthcare markets at increasing speed; these often cost more than current alternatives and significantly affect the cost of healthcare services and delivery (1). Identifying future technologies supports service preparedness, long-term planning, and strategic decision making. The aim of this study was to describe and classify health technologies predicted in fifteen forecasting studies according to their type, purpose and clinical use, and relate these to the original purpose and timing of the forecasting studies.

This was a descriptive study of predicted healthcare technologies identified in fifteen forecasting studies included in a previously published systematic review (2). Outcomes related to (i) each forecast study including country, year, intent and forecasting methods used, and (ii) the predicted technology type, purpose, targeted clinical area and forecast timeframe.

We identified 896 predicted health-related topics, of which 685 were health technologies. Of these, 19.1 percent were diagnostic or imaging tests and 14.3 percent devices or biomaterials; 38.1 percent were intended to treat or manage disease and 21.6 percent to diagnose or monitor disease. The most frequent targeted clinical areas were infectious diseases followed by cancer, circulatory and nervous system disorders. The mean timeframe for technology forecast was 11.6 years (Standard Deviation, SD = 6.6). The forecasting timeframe significantly differed by technology type (p = .002), the intent of the forecasting group (p < .0001), and the methods used (p < .0001).

Our description and classification of predicted health-related technologies from prior forecasting studies provides an overview of the technological and clinical frontiers of innovation in health and healthcare provision.

Cost-effectiveness analysis is probably the most often applied analysis in the field of Health Technology Assessment. Yet, there are major issues related to this analytical approach. Apart from the discussions about discounting, and which type of costs should be included in the analysis, there is discussion about the measurement of the health effects. The dominant economic and decision-making research paradigm that underpins present protocols to value health show flaws and limitations.

All existing generic health instruments (for example,. EuroQol five dimensions questionnaire, EQ-5D; Health Utilities Index, HUI; Short Form, SF-6D) used to collect values for health states use a small, fixed set of health domains. Descriptions of health states are created based on these domains and their levels. Subsequently, these descriptions are valued with special preference-based methods (measuring the quality or importance of something). When developing those instruments, patients’ opinions were not actively incorporated in the choice of domains, nor have patients’ preferences been considered in assessing weights to the domains and their levels.

We developed a novel preference-based health measurement methodology that combines the strength of two existing measurement models for subjective phenomena: the discrete choice model and Rasch item response theory (1-3). This new approach is referred to as the multi-attribute preference response (MAPR) model. This is the first generic health preference-based model that is fully based on the perception and reporting from patients (experienced based) and is insensitive to adaptation mechanisms. Apart from being grounded on measurement theory, the valuation tasks are easy and attractive to perform in a self-completion setting. A first application based on this novel model has been worked out in a mobile app to measure the overall health condition of infants (reported by mothers).

Our recently introduced and informative health measurement model overcomes many problems associated with the conventional methods.

This study compared the National Institute for Health Research (NIHR) Health Technology Assessment (HTA) Programme portfolio of research with the united Kingdom (UK) burden of disease, as measured by Disability-Adjusted Life Years (DALYs).

Design: Cross-sectional study.

Setting: The HTA Programme cohort included all funded applications (n = 363) received by the HTA Programme during the period 1 April 2011 to 31 March 2016. The sample contained primary research and evidence syntheses, all purely methodological studies were excluded since these are not comparable to the other study types.

Main Outcome Measure: Proportion of spend for each of the twenty-one Health Research Classification System (HRCS) health categories were compared with burden of disease in the UK calculated using 2015 DALY data from the Institute for Health Metrics and Evaluation (IHME) Global Health Data Exchange (GHDx).

The funded HTA Programme projects totalled about GBP397million research spend, which broadly reflected the UK DALY burden. Overall, there was less than 5 percent difference between the actual and predicted programme spend based on the burden of disease in the UK in most instances (seventeen out of the twenty-one HRCS Health Categories).

The largest categories of apportioned spend were Cancer (accounting for 12.1 percent of portfolio), and Mental Health (11.8 percent of portfolio) which particularly reflected the 9.8 percent burden of disease to the UK. Most notable deviations from DALY, where spend was lower than disease burden, were in the Cancer, Cardiovascular and Musculoskeletal categories; which may reflect the importance of other, notably charity, funding.

The HTA Programme spend broadly aligns with burden of disease as measured using DALYs. Discrepancies were expected owing to the programme remit and its approach to commissioning research to address market failure particularly in areas that are not already well supported by research charities or industry. Regular review of DALY data during research prioritisation and commissioning allows the HTA Programme to identify and address shortfalls in disease areas and to balance its portfolio.

Guidance for developing economic models recommend that model structure is carefully considered, and assumptions varied in sensitivity analysis (1). Models in smoking cessation have typically used cohort-level approaches, although recently discrete event simulations (DESs) have been developed (2). DESs allow additional flexibility such as modelling changing risk over time, and recurrent events. Our aim was to explore the impact of varying model structure and assumptions on the cost-effectiveness of smoking cessation programs.

We built a cohort state-transition model which related mortality to smoking status and considered the prevalence (based on smoking status) of five comorbidities associated with smoking, each of which has an associated cost and quality of life decrement. We additionally built a patient-level DES, using the Discretely Integrated Condition Event framework (3). The DES used the same data as the cohort model, except considering incidence for comorbidities rather than prevalence. We considered a population of smokers aged 16 years old and an intervention costing GBP827 on which 27 percent of people quit, compared with no treatment. We produced results using the two models for comparable scenarios, and ran additional scenarios considering different assumptions.

In the cohort model, the incremental cost-effectiveness ratio (ICER) for intervention versus no treatment was GBP4,000/quality-adjusted life year (QALY). In the DES, modelling mortality linked to smoker status produced an ICER of GBP1,000/QALY and modelling mortality linked to comorbidities produced an ICER of GBP6,000/QALY. In the DES with mortality linked to comorbidities, varying the relative risk of comorbidities with time since quitting gave an ICER of GBP3,000/QALY. Including relapse increased the ICER to GBP21,000/QALY.

The ICER for the smoking cessation program changes when model assumptions are varied, although the choice of DES versus cohort model appears to make a relatively small difference. Inclusion of relapse substantially changes the ICER, demonstrating the importance of long-term effects in economic models.

Postoperative delirium has been associated with poorer long term survival in Transcatheter aortic valve replacement (TAVR) and Surgival aortic valve replacement (SAVR) patients. However, its effect on hospitalization costs and length of stay in these populations has not been formally assessed.

Using the Medicare Provider Analysis and Review File, we retrospectively analyzed elderly (80 years of age and older) Medicare patients receiving TAVR and SAVR in the United States during the 2015 fiscal year. ICD-9-CM codes were used to identify postoperative delirium diagnoses. The incremental hospital resource consumption, measured as hospital cost and length of stay, was estimated for patients with postoperative delirium during their TAVR or SAVR index hospitalization. Multivariate regression models were used for the adjusted cost estimates controlling for patient demographics, comorbidities, and complications.

A total of 21,088 claims were available for analysis (12,114 TAVR and 8,974 SAVR). The mean age of the TAVR group was older compared to the SAVR group (87 versus 84; p < .001) and TAVR patients presented with a higher comorbidity burden (Charlson Index score 3.0 versus 2.1; p < .0001). TAVR patients experiencing postoperative delirium during the index hospitalization was 1.6 percent compared to 3.6 percent of surgical patients (p < .0001). For the overall cohort, the regression adjusted incremental cost of postoperative delirium was (USD15,592; p < .0001). Patients experiencing delirium also had significantly longer hospital length of stay (4.16 days; p < .0001). When stratified by treatment approach, the adjusted incremental cost was USD13,862 for TAVR (p < .0001) and USD16,656 for SAVR (p < .0001).

While infrequent, postoperative delirium significantly increased hospital cost and length of stay following transcatheter or surgical aortic valve replacement (AVR). Despite a significantly higher comorbidity burden, TAVR was associated with lower postoperative delirium rates compared to SAVR. Moreover, post-TAVR delirium may be associated with less resource consumption than post-SAVR delirium. Future studies should seek to determine whether general anesthesia avoidance in appropriately selected transfemoral TAVR patients can further decrease rates of delirium.

Hemophilia is a rare, inherited bleeding disorder affecting an estimated 400,000 people worldwide (1). Characterized by spontaneous bleeding and long-term, irreversible joint damage, persons with hemophilia are often limited in normal day-to-day activities, including work/school, and require comprehensive care at specialized treatment centers. With replacement therapies extending survival by decades and vastly improving quality of life (QoL), routine prophylaxis is considered the standard-of-care in developed countries. However, due to the cost of replacement factor, access to treatment remains a challenge, and increased scrutiny over funding has been augmented by growing demands on healthcare budgets (2). Thus, the hemophilia community shares a unified goal of objectively defining patient-centered value in hemophilia care.

Using a three-tiered outcomes hierarchy model initially described by Porter (3), an international, multidisciplinary panel of health economics outcomes researchers and hemophilia experts developed a value framework for decision makers to assess value of various healthcare interventions in hemophilia.

The three tiers for assessing value are: (i) Health status achieved/retained; (ii) Process of recovery; and (iii) Sustainability of health. Tier one measures survival, quality of life (QoL), and hemophilia-specific outcomes of bleeding frequency, musculoskeletal complications, and severe bleeds, as well as function/activity (that is, lifestyle impairment). Tier two measures time to initial treatment or recovery and time missed at education/work, as well as disutility of care (that is, inhibitor development, pathogen transmission/infections, orthopedic intervention, and venous access). Tier three measures avoidance of bleeds, maintenance of productive lives, and long-term health, while capturing long-term consequences of insufficient therapy or age-related complications. Applicability of the framework can be demonstrated in areas of healthcare delivery, treatment regimen, and innovation for new therapies.

This value framework represents an initial collaboration with stakeholders to define and organize an array of patient-centric outcomes of importance in hemophilia into a practical tool that can influence treatment and funding decisions in hemophilia care.

Re-purposing of established products (EPs) – defined as marketed for 8 years or more – may represent a high value for patients and society. It has been recognized by the European Commission as an important factor contributing to greater access to new therapies. Due to a lower development cost, it could also represent a cost-effective alternative and help to reduce pressure on healthcare budgets. However, it is perceived that no financial incentives exist for the pharmaceutical industry to invest in new indications for EPs. The objective of this research was to review current European regulations and propose strategies stimulating development in this field.

We performed a targeted literature review and held two international expert panel workshops to discuss current policies and their implications, and issue recommendations for changes.

Within the current regulatory framework EPs face price cuts due to generic competition, reference pricing (RP), price re-negotiations or systematic price cuts, after a period of marketing presence. Extension of indications does not permit to increase or maintain the price. Generic substitution regardless of indication poses another challenge. Limited incentives in the form of an additional year of market protection exists only for new indication(s) registered within the first 8 years following initial approval. The expert panel proposed several strategies to stimulate development in this field, including: (i) extending the period in which registering a new indication results in additional market protection beyond 8 years and extending the duration of additional market protection; (ii) delaying inclusion in RP for EPs with a new value adding indication; (iii) establishing a differential pricing by indication; (iv) preventing temporarily generic substitution when an EP is prescribed for a new indication.

Current regulations represent a serious disincentive to develop new indications for EPs. Regulatory and pricing policy changes are needed to stimulate development in this important field.

The National Institute for Health Research Health Technology Assessment (NIHR HTA) Programme commissions research to inform health services in the United Kingdom. The program prioritises research ideas from literature, guidelines, patients, and clinicians, to decide which research should be funded. We get clinical input on these ideas through (i) committees of clinicians and patients and (ii) seeking written advice from multiple clinicians — a refereeing process. Chairs of our committees suggested that the material we sent to clinicians was too extensive and the method of response too burdensome. We set out to determine whether reducing the information provided or burden of response would improve the engagement of clinicians with our processes, and hence improve the quality of advice provided, and the research available to health services.

We undertook a factorial randomized controlled trial (University of Southampton Faculty of Medicine Ethics Committee #8192, Trial registration: ACTRN12614000167662). Each participant was randomized to receive one of two types of material to comment on, and one of two means to respond. In the first allocation participants were randomised in a 1:1 ratio between receiving a ‘vignette’ (a briefing paper of up to ten pages discussing possible research = usual practice), or a ‘commissioning brief’ (a single page summarising the proposed research). In the second allocation, the method of response was randomized, between a structured form and free text email.

We randomized 460 clinical experts, and 356 (77.4 percent) responded. The responses were graded for quality on a scale of 0 to 4 (higher scores better). Non-response was scored as 0. Analysis using ANOVA gave results of a structured response scoring .34 points (Standard Deviation, SD .36) over a freeform response (p = .02); and the commissioning brief as .04 points over a vignette (p = .81).

This was the first randomized trial to take place inside the secretariat of the HTA program. The difference in quality score between the brief and the vignette allocations was neither statistically nor practically important. The difference between the structured and freeform response was statistically significant, and sufficiently large to be important in practice. While the choice of material to share with clinicians seems unimportant we have shown that it is worth sending a structured response form to experts.

Understanding cost drivers and estimating societal costs are important challenges for economic evaluation of health technologies in low-and-middle-income countries (LMICs) (1). This study assessed community experiences of health resource utilization and perceived cost drivers from a societal perspective to inform the design of an economic model for the Community Level Interventions for Pre-eclampsia (CLIP) trials (2).

Qualitative research was undertaken alongside the CLIP trial in two districts of Sindh province, Pakistan. Nine focus groups were conducted with a wide range of stakeholders, including pregnant women, mothers-in-law, husbands, fathers-in-law, healthcare providers at community and health facility-levels, and health decision-/policy-makers at the district-level. The societal perspective included out-of-pocket (OOP), health system, and program implementation costs related to CLIP. Thematic analysis was performed using NVivo software.

Most pregnant women and male decision makers reported a large burden of OOP costs for in- and out-patient care, informal care from traditional healers, self-medication, childbirth, newborn care, transport to health facility, and missed wages by caretakers. Many healthcare providers identified health system costs associated with human resources for hypertension risk assessment, transport, and communication about patient referrals. Health decision-/policy-makers recognized program implementation costs (such as the mobile health infrastructure, staff training, and monitoring/supervision) as major investments for the health system.

Our investigation of care-seeking practices revealed financial implications for families of pregnant women, and program implementation costs for the health system. The societal perspective provided comprehensive knowledge of cost drivers to guide an economic appraisal of the CLIP trial in Sindh, Pakistan.

In the past decade numerous efforts have been made to enhance quality of care in the province of Québec for patients with ST-elevation myocardial infarction (STEMI). Despite two prior field evaluations and diffusion of a systematic review as well as recommendations, a third audit revealed persistent gaps in care, specifically excessive treatment delays. Our cardiovascular evaluation unit thus aimed to develop a more comprehensive quality improvement framework that further engaged healthcare professionals.

A literature update identified best practices and ways to reduce treatment delays and improve outcomes. This review, combined with the latest evaluation results, was used to establish structural and process quality standards adapted to the Québec context, via a consensus process with a panel of clinical experts. The standards identified quality-of-care targets and key elements of a governance structure to guide the improvement process. Quality indicators to monitor change were also developed. An implementation plan was then created, likewise based on literature and evaluation results.

For the first time, the unit publicly disseminated the results of the third evaluation according to region, in addition to standard individual hospital “report cards”. A summit conference was held during which the standards and indicators were presented to clinicians and other stakeholders, in collaboration with the health ministry and a panel of cardiovascular experts. Site visits are planned to facilitate change and establishment of local improvement plans and committees. A “tool kit” was developed containing a treatment algorithm, a drug protocol, five quality indicators each for processes and care networks, and measurement tools for indicators. A 75 percent minimal achievement target was set for treatment times.

A comprehensive framework aimed at improving quality of care for STEMI patients and monitoring change was created by combining evidence from the literature and “real world” data and mobilizing key stakeholders.

The adoption and reimbursement of a new or novel medical device frequently occurs after an economic evaluation of the innovation. One important factor for reimbursement rejections by the English National Institute for Health and and Care Excellence (NICE) Medical Technologies Evaluation Programme (MTEP) appears to be the little or no attention to early assessment (1). The aim of this study is to achieve a more in-depth and comprehensive understanding of the value of early Health Technology Assessment (HTA) for new medical devices.

This study employs a mixed methods research strategy. Our informant interviews involved two types of key stakeholders: health economists in academia and professionals in medical devices firms with a professional role in research and development or market access departments. Our qualitative analysis focused on two samples from six universities (five in the United Kingdom, UK, and one in Italy) and six small to medium-sized enterprises (five in the UK, and one in Italy). Insights from field work interviews helped to design our complementary quantitative analysis.

During thematic analysis, barriers to adoption of early HTA emerged across three domains. First, educational barriers (that is, what HTA/early HTA is and how to conduct it) influenced the foundation for the reimbursement strategy. Second, interviewees highlighted the presence of intrinsic barriers (for example, resources for translational and early preclinical research, reliability and reproducibility, evidence, and dissemination of sensitive information) within existing practices and knowledge. Third, several research gaps (that is, medical device classification, standardization of methods, guidelines for developers, and alignment of stakeholders perspectives) were identified. Finally, academics adopted early HTA to assess different aspects of a medical device early in development; however, developers were focused on the assessment of investment and safety/usability factors, especially for in-house evaluations.

If decision makers expect developers to produce better quality evidence and society aims to optimize resources that is, not investing in non-cost-effective technologies, then the incorporation of a more robust analytical framework including a societal perspective is necessary to understand how early HTA can be embedded into all aspects of the development process.

Patient-reported outcome measures (PROMs) provide a way to measure the impact of a disease and its associated treatments on the quality of life from the patients’ perspective. The aim of this review was to identify PROMs that have been developed and/or validated in patients with carotid artery disease (CAD) undergoing revascularization, and to assess their psychometric properties and examine suitability for research and clinical use.

Eight electronic databases including MEDLINE and CINAHL were searched from inception to May 2015 and updated in the MEDLINE database to February 2017. A two-stage search approach was used to identify studies reporting the development and/or validation of relevant PROMs in patients with CAD undergoing revascularization. Supplementary citation searching and hand-searching reference lists of included studies were also undertaken. The Consensus-based standards for the selection of health measurement instruments (COSMIN) and Oxford criteria were used to assess the methodological quality of the included studies, and the psychometric properties of the PROMs were evaluated using established assessment criteria.

Six PROMs, reported in five studies, were identified: 36-Item Short Form Health Survey (SF-36), Euro-QoL-5-Dimension Scale (EQ-5D), Hospital Anxiety and Depression Scale (HADS), Dizziness Handicap Inventory (DHI), Quality of life for CAD scale by Ivanova 2015 and a disease-specific PROM designed by Stolker 2010. The rigour of the psychometric assessment of the PROMs were variable with most only attempting to assess a single psychometric criterion. No study reported evidence on criterion validity and test-retest reliability. The overall psychometric evaluation of all included PROMs was rated as poor.

This review highlighted a lack of evidence in validated PROMs used for patients undergoing carotid artery revascularization. As a result, the development and validation of a new PROM for this patient population is warranted in order to provide data which can supplement traditional clinical outcomes (stroke >30 days post-procedural, myocardial infarction and death), and capture changes in health status and quality of life in patients to help inform treatment decisions.

Globally, health systems are struggling with reliably appraising the safety and efficacy of rapidly changing digital health interventions whilst allowing useful innovations to be rapidly adopted. Assessment and regulation of the large number of health apps should be proportional to their clinical risk, but there is large uncertainty about suitable criteria to assess risk (1). We aimed to identify criteria for assessing clinical risks associated with different types of health apps.

Our work builds on previous studies that identified some of the risks that health apps can pose and contextual factors that can moderate these risks (2,3). This work is grounded in a review of existing literature; wide consultation of stakeholders; participation in multi-agency policy discussion; and sense-checking successive versions of the framework that evolved over time. We combined different risk domains for apps (technical safety, usability, intervention quality, and engagement) with their functions (learning, behaviour and cognition change, communication, record keeping, and clinical decision support).

We developed a comprehensive generic risk framework that app users, developers, commissioners, regulators and other stakeholders worldwide can use to guide assessment of the likely risks posed by a specified health app in a specific context. We also propose questions that should help determine whether these risks have been addressed.

Apps are very promising in health care but are very numerous, complex, rapidly evolving and with overlapping functions. A rigorous risk framework should help stakeholders to deal with the large quantity of health apps, classify and manage clinical risks, and improve patient safety by applying generic risk assessment criteria. Further work is needed to test and develop the criteria we propose, especially as apps that integrate different functions are emerging, which will make risk assessment more complex.

In Hong Kong, the Drug Advisory Committee (DAC) has had the role of evaluating and advising new drugs to be included in the listing of the Hospital Authority Drug Formulary since July 2005. The drug review process was subject to challenge due to a lack of transparency to members of the public and documentation of the scientific basis for decision making. The purpose of this review was to describe the process, evaluation criteria and possible outcomes of decision making for new drugs listed in the Hong Kong Hospital Authority Drug Formulary in comparison to Health Technology Assessment (HTA) policies in overseas countries.

Details of the decision-making processes including new drug listing submissions, the DAC meeting, procedures before and after the meeting, were extracted from the official Hong Kong Hospital Authority drug formulary management website and manual. Publicly available information related to new drug decision making processes for four HTA agencies (National Institute for Health and Clinical Excellence (NICE), Scottish Medicines Consortium (SMC), Australian Pharmaceutical Benefits Advisory Committee (PBAC), and Canadian Agency for Drugs and Technologies in Health (CADTH)) were reviewed and retrieved from official documents on their public domains.

The DAC is in charge of the systematical and critical appraisal of new drugs for listing on the formulary, reviewing submitted applications, and making decisions of drug listing based on scientific evidence in which safety, efficacy and cost-effectiveness are primary considerations. When compared to other HTA agencies, transparency of decision-making processes of the DAC, relevance of clinical and health economic evidence, and lack of health economic and methodological input to submissions were major challenges of the new drug listing policy in Hong Kong.

Despite the challenges identifed, this review provided suggestions for establishing a more transparent, credible, evidence-based decision-making process for the Hong Kong Hospital Authority Drug Formulary. Proposals for improvement in the listing of new drugs in the formulary should be a priority in healthcare reform.

As the Health Technology Assessment (HTA) community moves towards greater levels of Patient and Public Involvement (PPI), the need to communicate with the public about the results and impact of HTA, and of PPI itself, is also increasing. DECIDE was a European Commission funded collaborative project arising from the GRADE working group, which developed and evaluated strategies for effectively communicating the recommendations from clinical guidelines to a multiple stakeholders. The Scottish Intercollegiate Guidelines Network (SIGN) led the work stream on patients and the public (1, 2). We extended the findings to develop a patient/public version of an HTA on Antimicrobial Wound Dressings (AWDs). The clinical and cost effectiveness evidence was inconclusive (3) which increased the importance of engagement with clinical and patient/public stakeholders.

A literature review, and a series of focus groups and user testing informed the development of a set of principles for designing patient versions of guidelines (1,2). Using these principles developed by the DECIDE collaboration, a patient version of the HTA was developed, in partnership with public volunteers and a standing public communications advisory group.

We incorporated key facilitators of usability, such as distinct branding as material for the public, a clearly communicated purpose, and the layering of information. Other facilitators included a “friendly” and accessible tone which was achieved by the use of colour, icons, simple language and charts, and brief chunked text. While feedback about clarity, design and usefulness was generally positive, some public reviewers were concerned by the level of uncertainty and complexity in the findings.

Using the principles from the DECIDE project, it is feasible and useful to develop public versions of an HTA report. The patient version is currently informing the development of educational material for patients/public about chronic wounds and AWDs by the Scottish Government. However, it remains a challenge to balance the complexity and uncertainty underlying evidence-based recommendations, with the need to provide accessible, understandable, and yet accurate information about them for the public.

Involving members of the public in the development of Health Technology Assessments (HTAs) has scientific and public value (1) but the most common form of involvement in HTA remains collection of people's views in the form of data (2). Involving members of the public in shaping the research is rare due to perceived time or resource constraints (3). Our research aimed to; (i) develop tailored meeting formats for public involvement in a lung cancer screening HTA, (ii) capture views on lung cancer screening using a community drop in format (iii) explore how different groups of public contributors, with various prior experience of involvement, participated in the HTA consultation process.

The involvement included three separate public meetings and a drop-in session at a community centre. Meeting formats were specifically tailored to meet the needs of the lung cancer screening HTA while drawing on previous patient and public involvement (PPI) work in relevant disciplines. All meetings were audio recorded and observed using a structured form. This data is currently being analysed using a combination of inductive and thematic analysis.

The qualitative research data on PPI processes was collected in November 2016. The paper will present results from our full analysis. At present, we note that while limited time was available to explain HTA to participants, this did not hamper the discussions’ relevance to the HTA work. Participants shared personal stories irrespective of whether they knew each other from before. People drew on own and others’ experiences when discussing outcomes of importance to this HTA.

Prior involvement in research or specific research methods training may not be necessary for public involvement in HTA. This has implications for involving diverse or “hard to reach” groups, without high levels of associated cost.